Kaleido Biosciences presents data from clinical study of KB195
Kaleido Biosciences announced data from a clinical study of KB195 in patients with urea cycle disorders are being featured in a poster presentation during the Society for the Study of Inborn Errors of Metabolism Annual Symposium. Data presented at SSIEM are from an open-label, single-arm, clinical study to evaluate the safety, tolerability and effect of KB195 on measures of nitrogen metabolism in patients with well-controlled UCD. The study was conducted at a single center and enrolled four patients who received KB195 orally for 21 days, while continuing their standard of care. In the study, no clinically significant safety signals were observed with KB195 and there were no serious treatment-emergent adverse events. KB195 was generally well tolerated based on measures that included the Gastrointestinal Tolerability Questionnaire, and the Bristol Stool Scale. Patients were well-controlled on their standard of care at study entry, and plasma ammonia levels were within the normal range at baseline and remained normal throughout the study. A lactose-15N-ureide tracer was used to evaluate changes in nitrogen metabolism in the gut. A reduction of this tracer in the urine and an increase of the tracer in the stool are consistent with a reduction in net ammonia production by the gut microbiome. While the study wasn't designed to demonstrate a significant effect on this biomarker of ammonia production, in evaluable patients, KB195 was associated with a general decrease in urinary 15N excretion and an increase in stool 15N excretion, suggestive of KB195 activity in reducing nitrogen metabolism. A global, multi-center, open-label, single arm Phase 2 clinical trial of KB195 is being conducted under an Investigational New Drug application in the U.S. and approved Clinical Trial Application in other countries. It is designed to evaluate the efficacy and safety of KB195 in UCD patients with elevated ammonia levels on standard of care. Patients enrolled in UNLOCKED will receive KB195 administered orally for eight weeks in addition to their standard of care, which includes dietary protein restriction and may include treatment with a nitrogen binding therapy.